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EP 4 240 427 A1

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(43) Date of publication: 13.09.2023 Bulletin 2023/37 (21) Application number: 21890271.6 (22) Date of filing: 09.11.2021

(51) International Patent Classification (IPC):  A61K 48/00(2006.01) C12N 15/861(2006.01) C12N 15/86(2006.01) (52) Cooperative Patent Classification (CPC): C12N 15/86; C12N 2750/14143; A61P 25/00; A61K 39/42; A61K 35/761; A61K 48/0075; A61K 2039/54; A61K 2039/5256; C07K 2317/76; C07K 16/081; A61K 2039/505; C07K 16/06   C-Sets: A61K 39/42, A61K 2300/00; (86) International application number: PCT/US2021/058572 (87) International publication number: WO 2022/099179 (12.05.2022 Gazette 2022/19)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 09.11.2020 US 202063111496 P 15.10.2021 US 202163256437 P (71) Applicants: Affinia Therapeutics Inc. Waltham, MA 02453 (US) Massachusetts Eye & Ear Infirmary Boston, MA 02114 (US) The Schepens Eye Research Institute, Inc. Boston, MA 02114 (US)

(72) Inventors: CALCEDO DEL HOYO, Roberto Waltham, MA 02453 (US) VANDENBERGHE, Luc, H. Boston, MA 02114 (US) (74) Representative: J A Kemp LLP  80 Turnmill Street London EC1M 5QU London EC1M 5QU (GB)